A Study of 5-Azacitidine (Vidaza®) in Patients With Chronic Myelomonocytic Leukemia

This study has been completed.

Sponsor:

University of Utah

ClinicalTrials.gov Identifier:

NCT01350947

First Posted: May 10, 2011

Last Update Posted: June 13, 2016

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
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Response to treatment will be evaluated according to the revised International Working Group (IWG) categories natural history, hematologic improvement and cytogenetic response1;2. The primary objective is:

To determine the rate of complete hematologic response and hematologic improvement (according to IWG 2006 criteria) in CMML patients treated with 5-azacitidine.

To provide a homogeneous suspension, the contents of the syringe must be re-suspended by inverting the syringe 2-3 times and vigorously rolling the syringe between the palms for 30 seconds immediately prior to administration.

The 5-azacitidine suspension is administered subcutaneously.

Intravenous Administration:

5-Azacitidine solution is administered intravenously. Administer the total dose over a period of 10-40 minutes.

Other Name: Vidaza®

Detailed Description:

In this study, eligible patients with a confirmed diagnosis of CMML will be treated with 5-azacitidine to determine the rates of complete hematologic response, hematologic improvement, complete and partial cytogenetic response, and overall and progression free survival.

To develop biomarkers associated with response and gain insights into the mechanisms that determine response, gene expression profiling, genome-wide SNP array analysis, microRNA analysis, and DNA methylation analysis will be performed prior to therapy and at defined time points during the study. Phosphoproteomics profiling may be included in the analysis.

Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:

18 Years and older (Adult, Senior)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Diagnosis of CMML as defined by the WHO criteria

Persistent peripheral blood monocytosis of more than 1 x 109/L for at least 3 months and

No Philadelphia chromosome or BCR-ABL fusion gene and

Less than 20% blasts in the blood or bone marrow and

Dysplasia in one or more of the myeloid lineages* * In the absence of dysplasia in one or more of the myeloid lineages, the diagnosis of CMML can still be made if a) - c) are met AND an acquired clonal chromosomal abnormality is present in the bone marrow cells, the monocytosis has been present for more than 3months AND all other causes of monocytosis have been ruled out.

Age of 18 years or older. Both men and women and members of all races and ethnic groups will be included.

ECOG performance status <3

Adequate organ function defined as:

Total bilirubin <2.5 x upper limit of normal (ULN)

Direct bilirubin <2 x ULN

Creatinine <2 mg/dL

ALT and AST <2.5 x ULN

Ability to understand and the willingness to sign a written informed consent document

Willingness to use adequate contraception for the duration of the study

Exclusion Criteria:

Progression to acute myeloid leukemia (defined by at least 20% blasts in the blood or bone marrow). In the unlikely event that progression to acute leukemia is demonstrated in the "screening" bone marrow biopsy, it is at the discretion of the investigator to enroll the patient after adequate discussion of the findings and alternative therapies. Enrollment of such a patient must be reported to the HCI PI.

Presence of activating mutations of the platelet derived growth factor receptors alpha or beta, which would suggest likely benefit from imatinib treatment (these mutations will usually be obvious from karyotyping and fluorescence in situ hybridization studies)